His Highness Sheikh Mohammed bin Rashid Al Maktoum, Vice-President and Prime Minister of the UAE and Ruler of Dubai has generously funded the treatment of a 19-month-old Iraqi girl which is worth AED8 million.
The gesture will provide the Iraqi baby with a complete reversal of her genetic condition, which is dubbed as the world’s most expensive gene therapy.
The patient, Laveen Jabbar Al Kutyashi, was diagnosed with Stage II Spinal Muscular Dystrophy (SMA), a genetic condition that delays motor development. According to science, without this treatment, her condition would have progressively deteriorated.
Laveen’s mother posted a video on social media, appealing for help which was viewed by the Dubai ruler.
“Luckily for the child, when the family came here from Iraq as directed by His Highness Sheikh Mohammed, she was diagnosed at our centre for excellence for genome studies to be suffering from this genetic condition. Al Jalila Children’s Speciality Hospital is the first and only one hospital in the entire region to provide this remarkable gene therapy for SMA,” Dr. Mohammad Al Awadhi, CEO of Al Jalila Children’s Speciality Hospital, told Gulf News.
The gene therapy, a one-time injection, for this condition has already been approved by the Food and Drug Authority USA three years ago. Through this treatment, Laveen will be administered with an infusion of the drug Zolgensma (AVXS-101) according to her body weight.
“The earlier this drug is given, the better it is as it stops the muscular degeneration. In this case, the little girl was recently diagnosed, so the prognosis for her is very good,” Dr. Al Awadhi.
Once the drug is administered, Laveen will be monitored by the pediatricians and will also be put through a rehabilitation program.
“When I got a call from Al Jalila Hospital, my wife and I couldn’t believe that the Ruler of Dubai would make such a gesture to us. You cannot imagine our happiness and the hope that this has given us. Without this, we would have lost our baby,” Laveen’s father, Ibrahim Jabbar Mohammad, said.
According to Laveen’s parents, she was born completely fine and had normal movements, however, after turning six months, her movements had suddenly stopped.
She was suspected Stage II SMA. “However, it was diagnosed only in October 2019 when we were able to travel to Turkey after the lockdown for the pandemic was lifted. Genetic tests carried out there confirmed our worst fears and we were in great despair,” the parent said.
“My daughter has a new lease of life, thanks to the generosity of Sheikh Mohammed,” they added.